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Charcot-Marie-Tooth Disease clinical trials

The registry lists 113 studies for Charcot-Marie-Tooth Disease across more than 10 countries. 37 are recruiting or about to start. 76 of them are mainly about Charcot-Marie-Tooth Disease (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-12.

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Status

Completed
51
Recruiting
31
Active, not recruiting
8
Status unknown
8
Not yet recruiting
6
Terminated
6

Phase

Phase 2
16
Phase 1
7
Phase 3
6

Where

United States
41
France
23
Italy
11
United Kingdom
11
Turkey
10
Canada
9

Who runs them

University Medical Center Goettingen (5) · University of Iowa (4) · Pharnext S.C.A. (4) · Centre Hospitalier Universitaire de Liege (3) · Peking University Third Hospital (3) · Assistance Publique Hopitaux De Marseille (3) · University Hospital, Clermont-Ferrand (3) · Ankara City Hospital Bilkent (3)

What is being tested here

Drugs under test in Charcot-Marie-Tooth Disease studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted

DrugOpenPhase 3All
EN001102
Cannabidiol101
Epalrestat101
PXT3003023
Ascorbic Acid012
Efmitermant alfa002
Baclofen011
Naltrexone011
Sorbitol011
Ignaseclant001

Coming up

2phase 3 studies under way
16studies finish main data collection within a year

Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.

The 10 nearest

Who these studies take

Of the 31 Charcot-Marie-Tooth Disease studies recruiting now, 14 (45%) set no upper age limit and 13 (42%) stop at 75 or younger. 14 (45%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 13 (42%) take healthy volunteers.

Recruiting studies that take each age, of 31
AgeWomenMen
1013 (42%)13 (42%)
1827 (87%)27 (87%)
3029 (94%)29 (94%)
4028 (90%)28 (90%)
5028 (90%)28 (90%)
6525 (81%)25 (81%)
7519 (61%)19 (61%)
8516 (52%)16 (52%)

Across all 113 studies, recruiting or not: 43% stop at 75 or younger and 32% at 65; 38% take children; 1% are for women only and 0% for men only; 30% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.

How long Charcot-Marie-Tooth Disease studies take

Of 38 interventional studies with no phase under Charcot-Marie-Tooth Disease, half reached primary completion within 29 months of starting, counting those still running.

Start to primary completionEvery sponsorIndustryNon-industry
Interventional, no phase29 mo7–67 · 38 studies—29 mo7–67 · 38 studies

Each figure is the month by which half the studies had reached primary completion, with the range in which the middle half did. Studies still running count until their last registry update; studies that stopped early are left out. Phase 1 includes early phase 1; observational means prospective or not stated, as retrospective and cross-sectional studies date a window of data rather than a study. How these figures are worked out

What changed recently

From the registry's own updates since Oct 14, 2026.

Newly registered1

Studies

Recruiting first, then most recently updated. Showing 50 of 113. Browse all 113 → or search them.